Endpoint Enabling Study in CDK-like 5 Deficiency Disorder (CDD)
Research type
Research Study
Full title
Endpoint Enabling Study in Cyclin-dependent kinase-like 5 Deficiency Disorder (CDD)
IRAS ID
315263
Contact name
Sam Amin
Contact email
Sponsor organisation
loulou foundation
Duration of Study in the UK
3 years, 9 months, 26 days
Research summary
This is a multicenter, multinational study which will be conducted in the US, UK and the EU with 40 Sites will recruit approximately 100 Patients from the ages 0 to 55, with Cyclin-dependent kinase-like 5 (CDKL5) Deficiency Disorder (CDD) caused by a genetic error. Patient with CDD suffer from serious symptoms which include seizures and epilepsies. No Study drug or Placebo will be administered for the patients in this study. This study will follow individuals with confirmed CDD over the course of 3 years. Study procedures will include in-clinic evaluations at Day 1 and months 6, 12, 18, 24 and 36 months. At the in-clinic visits, an examination will include evaluating the head, eye, ear, nose, and throat, and the cardiovascular, skin, musculoskeletal, respiratory, gastrointestinal, and neurological systems. The remote assessments at 3, 9 and 30 months. No Blood samples will be taken throughout the trial. The patient/Carer will be asked to complete various questionnaires aimed at the CDD population with the goal of helping the sponsor to collect important information about the condition to develop better designed future clinical trials. Throughout the duration of the entire study, the patient/carer will also be required to complete a Seizure Diary.
REC name
Wales REC 4
REC reference
22/WA/0186
Date of REC Opinion
6 Sep 2022
REC opinion
Further Information Favourable Opinion